Breakthrough Therapy Designation granted to Rina-S for the treatment of adult patients with recurrent or progressive endometrial cancer (EC) who have disease progression on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy Regulatory decision supported by data from the Phase 1/2 RAINFOL™-01 trial showing encouraging responses in heavily pretreated EC patientsi Rina-S continues to be evaluated as a...Read more
Breakthrough Therapy designation complements Rusfertide's Orphan Drug and Fast Track designations, which together confer multiple benefits to the development program U.S. New Drug Application filing for Rusfertide in polycythemia vera on track for Q4 NEWARK, CA / ACCESS Newswire / August 25, 2025 / Protagonist Therapeutics, Inc. ("Protagonist" or "the Company") announced today that rusfertide, a potential first-in-class...Read more
Data from IDeate-Lung01 trial will be presented at upcoming IASLC 2025 World Conference on Lung Cancer First Breakthrough Therapy Designation for Daiichi Sankyo and Merck’s ifinatamab deruxtecan based on IDeate-Lung01 phase 2 trial, with support from IDeate-PanTumor01 phase 1/2 trial Fourteenth Breakthrough Therapy Designation granted by FDA across the oncology portfolio of Daiichi Sankyo BASKING RIDGE, N.J. & RAHWAY, N.J. / Aug...Read more
First Breakthrough Therapy Designation in the U.S. for SystImmune and Bristol Myers Squibb's Izalontamab brengitecan (Iza-bren) based on data from the BL-B01D1-101 (CN), BL-B01D1-203 (CN), and BL-B01D1-LUNG-101 (U.S.,/EU) studies REDMOND, Wash. and PRINCETON, N.J., Aug. 18, 2025 /PRNewswire/ -- SystImmune Inc. (SystImmune), a clinical-stage biotechnology company, and Bristol Myers Squibb (NYSE: BMY) today announced that the U.S....Read more
Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 WALTHAM, Mass., Aug. 04, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the U.S....Read more
Based on MATTERHORN Phase III trial results which demonstrated a statistically significant and clinically meaningful event-free survival benefit If approved, this will be the first and only perioperative immunotherapy-based regimen in this setting WILMINGTON, Del. / Jul 28, 2025 / Business Wire / AstraZeneca’s supplemental Biologics License Application (sBLA) for IMFINZI® (durvalumab) has been accepted and granted Priority Review in...Read more
On track for planned BLA submission for del-zota at year end 2025 SAN DIEGO, July 23, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota)...Read more
Breakthrough Therapy Designation granted to elironrasib for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer who have received prior chemotherapy and immunotherapy but have not been previously treated with a KRAS G12C inhibitor Designation based on encouraging clinical data observed with elironrasib in patients with advanced KRAS G12C non-small cell lung cancer REDWOOD...Read more
The Breakthrough Therapy Designation (BTD) was granted based on the highly compelling results from the Phase 2 EMBOLD trial in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) The EMBOLD cohort 2 pivotal trial is on track for topline results in H1 2026 with NDA filing to follow Praxis has recently initiated the EMERALD study investigating relutrigine broadly in DEEs BOSTON, July 17, 2025 (GLOBE NEWSWIRE) --...Read more
Breakthrough Therapy designation based on interim clinical data from Phase I/II trials showing clinically meaningful improvements in cardiac biomarkers and functional measures LX2006 also selected for FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, created to facilitate CMC registrational readiness and support faster patient access NEW YORK, July 07, 2025 (GLOBE NEWSWIRE) -- Lexeo...Read more
Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment...Read more
Breakthrough Therapy Designation based on promising early clinical evidence observed with daraxonrasib in patients with pancreatic ductal adenocarcinoma (PDAC) RASolute 302, a Phase 3 registrational study of daraxonrasib in patients with previously treated metastatic PDAC, expected to substantially complete enrollment this year REDWOOD CITY, Calif., June 23, 2025 (GLOBE NEWSWIRE) -- Revolution Medicines, Inc. (Nasdaq: RVMD), a...Read more
Based on Type C meeting and new data, Dyne submitted revised ACHIEVE trial protocol to FDA elevating vHOT to primary endpoint for U.S. Accelerated Approval New positive clinical data from Phase 1/2 ACHIEVE trial support vHOT as early indicator of clinical benefit with DYNE-101 in DM1 Ongoing Registrational Expansion Cohort in ACHIEVE trial to enroll 60 participants and include sites in U.S. Company to host an...Read more
Designation Supported by CLOVER WaM Phase 2 Study Data Which Reported an 83.6% Overall Response Rate (ORR) Seeking Guidance from EMA to Determine if CLOVER WaM Phase 2 Data Meets Criteria to Apply for Fast-Track, Conditional Marketing Authorization, Answer Expected Late July FLORHAM PARK, N.J., June 04, 2025 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on...Read more
Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant’s efficacy and favorable safety profile Trial completion expected by Q4 2025 (including a three-month follow-up period) Tinlarebant has previously been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and the Pioneer Drug Designation in Japan for...Read more
Breakthrough Therapy Designation request based on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response Veligrotug met all of its primary and secondary endpoints in the pivotal THRIVE and THRIVE-2 clinical trials in active and chronic TED First and only drug candidate in chronic TED to demonstrate statistically significant and clinically...Read more
Registrational study in patients with tumor hyperinsulinism (HI) expected to commence mid-year Designation underscores need for therapies to treat severe hypoglycemia in the oncology setting REDWOOD CITY, Calif., May 05, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage rare disease company focused on treating hypoglycemia caused by HI, today announced that the U.S. Food and Drug...Read more
Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025 LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative...Read more
Designation enables expedited development and priority regulatory review BTD application was supported by updated clinical data from Ph2 neoadjuvant UM trial that we are targeting to present at medical conferences in mid-2025 and H2 2025 Targeting to initiate a Ph3 registrational study in neoadjuvant UM in H1 2025 Neoadjuvant UM has a projected annual incidence of ~12k patients, and is a high unmet medical with no FDA approved...Read more
UTRECHT, The Netherlands and CAMBRIDGE, Mass., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics®, Triclonics® and ADClonics®), for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to petosemtamab in combination with...Read more
Designation is based on TELLOMAK Phase 2 results demonstrating efficacy and a favorable safety profile in patients with advanced Sézary syndrome heavily pre-treated, post- mogamulizumab. Breakthrough Therapy Designation is intended to accelerate the development and regulatory review in the U.S. of drugs that are intended to treat a serious condition; adding to a Fast Track designation by the U.S. FDA received in 2019 as well as a PRIME...Read more
The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets Adaptimmune Allo-T program to be featured at the Biotech ShowCase(TM) and the Wuxi Global Forum 2025 Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine...Read more
SOUTH SAN FRANCISCO, Calif., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all...Read more
Breakthrough Therapy Designation granted based on key positive data from the Phase 2b (RIZE) study and current unmet medical need in congenital hyperinsulinism (HI) Ersodetug continues to advance in clinical development as a potential treatment for hypoglycemia caused by all forms of hyperinsulinism; topline sunRIZE data expected second half of this year REDWOOD CITY, Calif., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq:...Read more
FOSTER CITY, Calif. / Dec 17, 2024 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy. The Breakthrough Therapy Designation is...Read more
Designations aim to expedite the development and review of promising therapies for serious conditions with unmet medical needs Phase 3 ECLIPSE registrational program in chronic hepatitis delta to begin in the first half of 2025 SAN FRANCISCO / Dec 12, 2024 / Business Wire / Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that tobevibart and elebsiran have received U.S. Food and Drug Administration (FDA) Breakthrough...Read more
First Breakthrough Therapy Designation for AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan Based on TROPION-Lung05 Phase II trial and supported by data from TROPION-Lung01 Phase III trial WILMINGTON, Del. / Dec 09, 2024 / Business Wire / Datopotamab deruxtecan (Dato-DXd) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with locally advanced or metastatic epidermal growth...Read more
Designation based on encouraging Phase 1b clinical data, including that SER-155 resulted in a 77% relative risk reduction in bacterial bloodstream infections versus placebo Breakthrough Therapy meeting with FDA on next study of SER-155 in allo-HSCT expected in Q1 2025 Seres seeking SER-155 strategic partnership to accelerate next study in allo-HSCT and expand to multiple target populations CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE...Read more
Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies Update on the company’s plans for a global, randomized, controlled Phase 3 registrational study anticipated by year-end BEDFORD, Mass. / Dec 04, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by...Read more
First breakthrough therapy designation for investigational sac-TMT in the U.S. RAHWAY, N.J. / Dec 03, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sacituzumab tirumotecan (sac-TMT) for the treatment of patients with advanced or metastatic nonsquamous non-small cell lung...Read more
WOBURN, Mass., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today announced that it has submitted a biologics license application (BLA) to the FDA for RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of adult patients with advanced melanoma who have previously received an anti-PD1...Read more
VAX-31 Infant Indication: Investigational New Drug Application Cleared by FDA; Company Expects to Initiate VAX-31 Infant Phase 2 Study by the End of January 2025 VAX-31 Adult Indication: Breakthrough Therapy Designation Granted by FDA; Company Plans to Initiate Adult Phase 3 Pivotal, Non-Inferiority Study by Mid-2025 VAX-31, Designed to Cover Currently Circulating and Historically Prevalent Strains, is Being Studied for the...Read more
The Breakthrough Therapy Designation (BTD) for investigational nipocalimab in Sjögren's disease, a prevalent autoantibody disease with no approved advanced therapies, is supported by results from the Phase 2 DAHLIAS study A greater than 70 percent relative improvement in systemic disease activity at Week 24 was demonstrated in study participants on average who received nipocalimab 15 mg/kg compared to participants who received...Read more
Designation based on positive interim analysis of Phase 2b VANTAGE study FOSTER CITY, Calif. / Oct 10, 2024 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). The regulatory designation is based on the...Read more
Designation is based on data from the clinical development program which demonstrated clinical proof of concept Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development FDA Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or...Read more
NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type...Read more
Supported by positive data from Phase 2b FASCINATE-2 trial of denifanstat in patients with MASH Preparations are ongoing to initiate Phase 3 program for denifanstat by the end of 2024 SAN MATEO, Calif., Oct. 01, 2024 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today...Read more
U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome LA JOLLA, Calif. / Sep 19, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA has granted Rare...Read more
Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s) BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and...Read more
PALISADE Phase 3 results demonstrate plozasiran reduced triglycerides by 80% from baseline and reduced the risk of developing acute pancreatitis by 83% in patients with genetically confirmed and clinically diagnosed FCS1 PASADENA, Calif. / Sep 10, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to...Read more
SEATTLE and VANCOUVER, British Columbia, July 31, 2024 (GLOBE NEWSWIRE) -- Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine dependence, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for cytisinicline for nicotine e-cigarette, or vaping,...Read more
U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for bexicaserin for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs) LA JOLLA, Calif. / Jul 01, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced...Read more
BRISBANE, Calif., May 28, 2024 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to atacicept for the treatment of IgAN. The designation reflects the FDA’s...Read more
DURECT plans to confirm the efficacy and safety of larsucosterol in a registrational Phase 3 clinical trial CUPERTINO, Calif., May 21, 2024 /PRNewswire/ -- DURECT Corporation (Nasdaq: DRRX), a late-stage biopharmaceutical company pioneering the development of epigenetic therapies to transform the treatment of serious and life-threatening conditions such as acute organ injury and cancer, today announced that the U.S. Food and...Read more
The designation is based on Phase III INAVO120 results, showing the inavolisib-based regimen more than doubled progression-free survival compared with palbociclib and fulvestrant alone in the first-line setting Approximately 40% of people with HR-positive breast cancer have a PIK3CA mutation and often face poorer prognosis and resistance to endocrine treatment This is the 29th Breakthrough Therapy Designation for Genentech’s...Read more
CAMBRIDGE, Mass., May 16, 2024 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to NVL-655 for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell...Read more
Petosemtamab granted BTD for the treatment of previously treated HNSCC UTRECHT, The Netherlands and CAMBRIDGE, Mass., May 13, 2024 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for...Read more
Avidity initiating global Phase 3 HARBOR™ study for delpacibart etedesiran this quarter Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data SAN DIEGO, May 8, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA...Read more
First Ever Breakthrough Designation for a Drug Being Developed for PWS Designation is Based on Data from the Phase 3 Program for DCCR Planned Submission of a New Drug Application (NDA) for DCCR Remains on Track for Mid-2024 REDWOOD CITY, Calif., April 29, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare...Read more
Ziftomenib is the first investigational treatment to be granted Breakthrough Therapy Designation for NPM1-mutant AML Registration-directed trial of ziftomenib in NPM1-mutant AML on track to complete enrollment by mid-2024 SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment...Read more
Breakthrough Therapy Designation (“BTD”) provides an expedited review pathway, as well as increased access to U.S. Food and Drug Administration (“FDA”) guidance on trial design, with the potential to significantly reduce drug development timelines First known BTD granted by the FDA for an adjunctive psychedelic based therapy for the treatment of Major Depressive Disorder (“MDD”) Robust, sustained and statistically...Read more
A single oral administration of MM120 100 µg met its key secondary endpoint and maintained a clinically and statistically significant HAM-A reductions compared to placebo at 12 weeks with a 65% clinical response rate and 48% clinical remission rate MindMed plans to hold an End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA) in the first half of 2024 and initiate its Phase 3 clinical program in the second half of...Read more
CAMBRIDGE, Mass., Feb. 27, 2024 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to NVL-520 for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC)...Read more
Latozinemab is the most advanced progranulin-elevating candidate in development for FTD-GRN and has now become the first investigational medicine to receive a Breakthrough Therapy Designation for the treatment of FTD-GRN SOUTH SAN FRANCISCO, Calif., Feb. 07, 2024 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC) and GSK plc (LSE/NYSE: GSK) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough...Read more
Designation is based on Phase 1/2 safety and efficacy data in patients with Human Epidermal Growth Factor Receptor 2 (“HER2”)-expressing advanced endometrial cancer with encouraging early signs of anti-tumor activity Breakthrough Therapy designation will allow for an expedited development and regulatory review of BNT323/DB-1303 Endometrial or uterine cancer is the second most common gynecologic cancer globally with over 400,000 cases...Read more
Company Today Also Provided Updates on R&D Portfolio and Strategy at Investor Event Crinecerfont New Drug Application Submission Planned in 2024 NBI-'770, an Oral NMDA NR2B Negative Allosteric Modulator, Entering Phase 2 for the Treatment of Major Depressive Disorder Advancing Largest Portfolio of Muscarinic Compounds in Clinical Development Top-Line Phase 2 Data Readouts for Five Programs Anticipated in 2024...Read more
U.S. Food and Drug Administration (FDA) grants Breakthrough Therapy Designation (BTD) for epcoritamab-bysp for the treatment of relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy European Medicines Agency (EMA) validates regulatory application for epcoritamab for the same indication The regulatory actions are supported by data from the phase 1/2 EPCORE™ NHL-1 trial COPENHAGEN, Denmark /...Read more
Updates are supported by data from the Phase 1/2 EPCORE™ NHL-1 clinical trial NORTH CHICAGO, Ill., Nov. 27, 2023 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced updates from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for epcoritamab, an investigational T-cell engaging bispecific antibody administered subcutaneously, for relapsed or refractory (R/R) follicular lymphoma (FL). The FDA has...Read more
FDA granted Breakthrough Therapy Designation for felzartamab in PMN upon positive clinical data from M-PLACE, a Phase 2 study led by I-Mab partner HI-Bio I-Mab has full development and commercialization rights of felzartamab in Greater China for all indications, with Phase 3 multiple myeloma data expected in 2024, followed by a planned BLA submission ROCKVILLE, Md. and SHANGHAI, Nov. 2, 2023 /PRNewswire/ -- I-Mab (Nasdaq: IMAB) (the...Read more
Designation is based on results from the progressive pulmonary fibrosis cohort of the Phase 2 study assessing the safety and efficacy of BMS-986278 treatment versus placebo PRINCETON, N.J. / Oct 24, 2023 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for BMS-986278, a potential first-in-class, oral, lysophosphatidic acid...Read more
Supported by positive data in F2/F3 and F4 NASH patients from the ENLIVEN Phase 2b trial of pegozafermin Discussions with regulatory agencies regarding the NASH Phase 3 program planned for the fourth quarter of 2023 SAN FRANCISCO, Sept. 21, 2023 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver...Read more
Designation based on clinical evidence indicating INO-3107 may demonstrate substantial improvement over existing therapies First Breakthrough Therapy designation for an INOVIO DNA medicine candidate PLYMOUTH MEETING, Pa., Sept. 7, 2023 /PRNewswire/ -- INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer,...Read more
LEXINGTON, Mass., July 20, 2023 (GLOBE NEWSWIRE) -- T2 Biosystems, Inc. (NASDAQ:TTOO), a leader in the rapid detection of sepsis-causing pathogens and antibiotic resistance genes, announced today the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device designation for the Company’s Candida auris (C. auris) direct-from-blood molecular diagnostic test. This marks the third T2 Biosystems’ product to receive FDA...Read more
Zeno granted BTD for the treatment of NRG1+ non-small cell lung cancer UTRECHT, The Netherlands and CAMBRIDGE, Mass., July 05, 2023 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for...Read more
UTRECHT, The Netherlands and CAMBRIDGE, Mass., June 29, 2023 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for zenocutuzumab (Zeno) for the treatment of patients with advanced unresectable...Read more
First FDA Breakthrough Therapy Designation granted for AdenoVerse immunotherapy platform; designation prioritizes PRGN-2012 as a potential treatment for RRP Designation based on positive Phase 1 clinical data that showed 50% of patients were "surgery-free" (Complete Response) after PRGN-2012 treatment with a minimum follow up of 12 months post-treatment GERMANTOWN, Md. , June 20, 2023 /PRNewswire/ -- Precigen, Inc....Read more
CONSHOHOCKEN, Pa., April 18, 2023 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), today announced that resmetirom has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with NASH with liver fibrosis. The Company also announced that the...Read more
Designation based on positive data from Phase 2b KEYNOTE-942/mRNA-4157-P201 trial CAMBRIDGE, MA and RAHWAY, NJ / ACCESSWIRE / February 22, 2023 / Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, and Merck (NYSE:MRK), known as MSD outside of the United States and Canada, today announced that mRNA-4157/V940, an investigational personalized mRNA cancer vaccine, in...Read more
Designation based on positive topline data from the ConquerRSV Phase 3 pivotal efficacy trial, which demonstrated vaccine efficacy of 83.7% against RSV lower respiratory tract disease, defined by 2 or more symptoms in older adults CAMBRIDGE, MA / ACCESSWIRE / January 30, 2023 / Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced mRNA-1345, an investigational...Read more
Breakthrough Therapy Designation for 24-Valent Investigational Pneumococcal Conjugate Vaccine Candidate Based on Positive Topline Proof-of Concept Data Results in Adults Aged 18-64 That Suggest Potential Best-in-Class Profile Topline Safety, Tolerability and Immunogenicity Data from VAX-24 Phase 2 Study in Adults 65 and Older Expected in Q2 2023 Company Continues to Advance PCV Franchise, Including VAX-24 and VAX-31, While...Read more
U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for adagrasib (KRAZATITM) in combination with cetuximab in patients with KRASG12C-mutated advanced colorectal cancer (CRC) whose cancer has progressed following prior treatment with chemotherapy and an anti-VEGF therapy. Journal publication marks second New England Journal of Medicine (NEJM) inclusion in 2022 for adagrasib. SAN DIEGO, Dec. 21,...Read more
Revumenib is the first and only investigational treatment for R/R KMT2Ar acute leukemia to receive Breakthrough Therapy Designation Designation is based on Phase 1 data from the AUGMENT-101 trial that showed a 27% CR/CRh rate in KMT2A patients treated at RP2D Company remains on track to submit an NDA for revumenib by the end of 2023 with the potential for an expedited approval with a broad indication WALTHAM, Mass., Dec. 5, 2022...Read more
vTv Therapeutics Inc. (Nasdaq: VTVT) a clinical-stage biopharmaceutical company focused on the development of orally administered treatments for type 1 diabetes and psoriasis, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for TTP399 as an adjunctive therapy to insulin for the treatment of type 1 diabetes. TTP399 is a novel, oral, investigational once-daily...Read more
Recursion Pharmaceuticals is a clinical stage TechBio company leading the space by decoding biology to industrialize drug discovery. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously expands one of the world’s largest....
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